Pfizer Expands Rare Disease R&D

Pfizer is expanding its rare disease R&D activities through the establishment of a gene therapy platform to investigate potential treatments. Under an agreement with Spark Therapeutics, the companies will collaborate to progress the clinical program for SPK-FIX, a program incorporating a bio-engineered AAV vector for the potential treatment of hemophilia B, which is expected to enter Phase 1/2 trials in 1H15.
 
Spark will be responsible for clinical development through Phase 1/2 studies. Pfizer will assume responsibility for further studies, any regulatory approvals and global commercialization of the product.
 
Additionally, Pfizer has appointed Michael Linden, Ph.D., professor at King’s College London and director of the University College London Gene Therapy Consortium, to serve a two-year period leading gene therapy research in rare diseases.
 
“The fundamental understanding of the biology of hereditary rare diseases, coupled with advances in the technology to harness disarmed viruses as gene delivery vehicles, provide a ripe opportunity to investigate the next wave of potential life-changing therapies for patients,” said Mikael Dolsten, M.D., Ph.D., president of Worldwide R&D at Pfizer. “By establishing our gene therapy capabilities, we hope to gain a deeper understanding of the mechanisms that could potentially bring true disease modification for those suffering from devastating hematologic and neuromuscular diseases.”
 
 “The establishment of a gene therapy group under the leadership of Professor Linden will help Pfizer explore the potential of this important technology that could possibly benefit patients living with serious diseases,” said Kevin Lee, Ph.D., senior vice president and chief scientific officer of Pfizer’s Rare Disease Research Unit. “Professor Linden brings to Pfizer his extensive expertise in AAV technology obtained from over 20 years working in the field.”